Huntington’s disease drug produces promising results in landmark trial

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DNA
A DNA double helix is seen in an undated artist's illustration released by the National Human Genome Research Institute, May 15, 2012. (REUTERS/National Human Genome Research Institute/Handout)

A groundbreaking drug trial for Huntington’s disease has produced promising results, showing signs that the progress of the genetic illness could be slowed down by the experimental drug.

The results are being lauded as a landmark achievement, as this is the first time that any drug has shown a capability to suppress the devastating effects of the disease on brain cells.

Currently, medications and treatments for Huntington’s disease patients only deal with the symptoms and are unable to slow down the spread of the disease. As such, the groundbreaking results are of enormous importance to Huntington’s disease patients and their families.

Professor Sarah Tabrizi, director of University College London’s Huntington’s Disease Centre, led the phase one trial for the experimental drug. According to her, the results of the trial were “beyond what I’d ever hoped.”

Nancy Wexler, whose efforts contributed to the identification of the disease’s mutation in 1993, expressed excitement over the positive trial results.

I’m ecstatic,” said Wexler. “Huntington’s is horrible, one of the worst diseases known to mankind, and certain death… We know it’s a bad gene, making a bad protein, that makes people sick, that kills your brain cells. Anything that could impact that, we knew that that could be a cure.”

The excitement is shared by many in the scientific world because the experimental drug could potentially be adapted to suppress other forms of dementia and incurable brain disorders like Alzheimer’s and Parkinson’s diseases.

Swiss pharmaceutical company Roche has already invested $45 million in order to license the drug for clinical use.

Huntington’s disease is an incurable degenerative disease caused by a single gene defect that is passed down genetically. Patients suffering from the disease typically get symptoms in middle age. These symptoms include mood swings, anger and depression.

At later stages, patients develop uncontrolled movements, dementia and paralysis. Most patients suffering from the disease die within 10 years of being diagnosed.